Neuromyelitis Optica Spectrum DisordersNCT07526298
Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Study to Assess Disease Activity in Apparently Stable Patients
An observational study of Neuromyelitis Optica Spectrum Disorders, run by IRCCS San Raffaele at 1 site in Italy.
Trial summary
The present research is an observational clinical study. The project aims to investigate astrocytic damage, assessed through biological findings such as an increase in GFAP level and/or MRI water index, in patients with NMOSD and its potential role in predicting demyelinating relapses or affecting disability outcomes. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorders
- What is being tested?
- MRI and Immunological Factors
- How many people
- 50 participants
- Where
- 1 siteItaly
- Study dates
- 1 Apr 2026to 1 Apr 2027
- How long will I take part?
- 12 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study will include adult patients with NMOSD.
Who may be able to join
- Adult patients (age ≥18 years);
- NMOSD diagnosis with AQP4 IgG positive status assessed with a cell-based assay;
- Under rituximab treatment (standard of care) from at least 1 year and for not more than 5 years;
- Ability to provide written informed consent.
Who cannot join
- History of a clinical relapse or new/enlarging T2 lesion during the 6 months preceding the enrollment;
- Steroids treatment during 30 days before the enrollment;
- Any contraindication to MRI;
- Patients currently participating in a different clinical trial;
- Patients currently pregnant.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site
San Raffaele Hospital
Milan, Milan, 20132
Monica Margoni, MD, PhD, +39-02-26433032, margoni.monica@hsr.it
About this study
Official title: Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Multicenter Prospective Observational Study to Assess Disease Activity in Apparently Stable Patients
The present research is an observational clinical study. The project aims to investigate astrocytic damage, assessed through biological findings such as an increase in GFAP level and/or MRI water index, in patients with NMOSD and its potential role in predicting demyelinating relapses or affecting disability outcomes. It will also explore predictors of astrocytic relapses, focusing on demographic, clinical, and immunological biomarkers like T cell responses, B cell repopulation, and cytokine levels. The goal is to identify unrecognized disease activity, providing insights for future research and clinical trials.
The study will involve 8 sites in Italy: 6 NMOSD clinical centers for patient enrolment and 2 centers for bioengineering and biological analysis. Centralized analysis of the MRI images of all patients enrolled in the clinical centers will be performed by the Neuroimaging Research Unit Fase 1 of San Raffaele Hospital. A total of 50 patients will be included and they will be followed for 12 months.
Comprehensive evaluation of patients, including clinical assessment, bioengineering evaluation, MRI, and blood samples, will be conducted at baseline, month 6, and month 12. To assess silent astrocytic relapses, a specialized evaluation will take place at months 3 and 9, including clinical analysis, blood samples to assess biomarkers like GFAP, a reduced MRI protocol to assess MRI water index, and bioengineering evaluation. In the case of a classical relapse, a dedicated visit will occur within 5 days of symptom onset, using the same evaluation protocol as at months 3 and 9 (MRI and biomarkers will be evaluated if not done in the month before).
- Conditions
- Neuromyelitis Optica Spectrum Disorders
- Being tested
- MRI, Immunological Factors
- Keywords
- Relapse, Astrocytic damage, MRI, GFAP
- Medical subject headings
- Neuromyelitis Optica, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIDiagnostic test
MRI will be performed every 3 months from baseline to 1 year.
Immunological FactorsDiagnostic test
Immunological factors will be performed every 3 months from baseline to 1 year.
| Study group | Role | What they receive |
|---|---|---|
| Adult patients with NMOSD | Not described Receives: Diagnostic Test: MRI; Diagnostic Test: Immunological Factors |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identifying predictors of clinical relapse
The primary outcomes measures are: percentage of patients with relapse, defined by the occurrence of a novel/worsening NMOSD symptom (detected by patient reported outcomes or clinical judgment or bioengineering indexes) PLUS biological findings of astrocyte damage (increase of GFAP level and/or MRI water index).
Measured: From enrollment to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2026
- Study start1 Apr 2026 (estimated)
- Primary completion1 Apr 2027 (estimated)
- Study completion1 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Neuromyelitis Optica Spectrum DisordersStudies of the same condition, open and under way.
- More trials run by IRCCS San RaffaeleThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- IRCCS San Raffaele
- Sponsor type
- University, hospital or other
- Collaborators
- Amgen
- Responsible party
- Principal investigator, Prof. Massimo Filippi, IRCCS San Raffaele
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07526298
- Sponsor's protocol ID
- ISS 20247046 Uplitza
- Status last verified
- Feb 2026
- First submitted
- 6 Mar 2026
- Last updated
- 13 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Masuda H, Mori M, Hirano S, Uzawa A, Uchida T, Muto M, Ohtani R, Aoki R, Kuwabara S. Silent progression of brain atrophy in aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder. J Neurol Neurosurg Psychiatry. 2022 Jan;93(1):32-40. doi: 10.1136/jnnp-2021-326386. Epub 2021 Aug 6.
PubMed 34362853Margoni M, Gueye M, Meani A, Pagani E, Valsasina P, Storelli L, Preziosa P, Moiola L, Rocca MA, Filippi M. Substrates of 8.5-year clinical outcomes in aquaporin-4 IgG-positive neuromyelitis optica spectrum disorders. J Neurol. 2026 Feb 2;273(2):112. doi: 10.1007/s00415-026-13647-x.
PubMed 41627519Lorefice L, Carotenuto A, Fenu G. Silent burden: recognising and managing invisible symptoms in neuromyelitis optica. J Neurol Neurosurg Psychiatry. 2025 Jul 16;96(8):744-752. doi: 10.1136/jnnp-2025-336041.
PubMed 40506118Cacciaguerra L, Pagani E, Radaelli M, Mesaros S, Martinelli V, Ivanovic J, Drulovic J, Filippi M, Rocca MA. MR T2-relaxation time as an indirect measure of brain water content and disease activity in NMOSD. J Neurol Neurosurg Psychiatry. 2022 Apr 28:jnnp-2022-328956. doi: 10.1136/jnnp-2022-328956. Online ahead of print.
PubMed 35483915
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Multicenter Prospective Observational Study to Assess Disease Activity in Apparently Stable Patients
- ClinicalTrials.gov ID
- NCT07526298
- Acronym / sponsor ID
- ISS 20247046 Uplitza
- Conditions
- Neuromyelitis Optica Spectrum Disorders
- Keywords
- Relapse, Astrocytic damage, MRI, GFAP
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- IRCCS San RaffaeleUniversity, hospital or other
- Collaborators
- Amgen
- Study start
- 1 Apr 2026 (estimated)
- Primary completion
- 1 Apr 2027 (estimated)
- Study completion
- 1 Apr 2027 (estimated)
- First posted
- 13 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The present research is an observational clinical study. The project aims to investigate astrocytic damage, assessed through biological findings such as an increase in GFAP level and/or MRI water index, in patients with NMOSD and its potential role in predicting demyelinating relapses or affecting disability outcomes. It will also explore predictors of astrocytic relapses, focusing on demographic, clinical, and immunological biomarkers like T cell responses, B cell repopulation, and cytokine levels. The goal is to identify unrecognized disease activity, providing insights for future research and clinical trials.
The study will involve 8 sites in Italy: 6 NMOSD clinical centers for patient enrolment and 2 centers for bioengineering and biological analysis. Centralized analysis of the MRI images of all patients enrolled in the clinical centers will be performed by the Neuroimaging Research Unit Fase 1 of San Raffaele Hospital. A total of 50 patients will be included and they will be followed for 12 months.
Comprehensive evaluation of patients, including clinical assessment, bioengineering evaluation, MRI, and blood samples, will be conducted at baseline, month 6, and month 12. To assess silent astrocytic relapses, a specialized evaluation will take place at months 3 and 9, including clinical analysis, blood samples to assess biomarkers like GFAP, a reduced MRI protocol to assess MRI water index, and bioengineering evaluation. In the case of a classical relapse, a dedicated visit will occur within 5 days of symptom onset, using the same evaluation protocol as at months 3 and 9 (MRI and biomarkers will be evaluated if not done in the month before).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 12 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adult patients with NMOSD | – | Diagnostic Test: MRI, Diagnostic Test: Immunological Factors |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study will include adult patients with NMOSD.
Key inclusion criteria (4 listed)
- Adult patients (age ≥18 years);
- NMOSD diagnosis with AQP4 IgG positive status assessed with a cell-based assay;
- Under rituximab treatment (standard of care) from at least 1 year and for not more than 5 years;
- Ability to provide written informed consent.
Key exclusion criteria (5 listed)
- History of a clinical relapse or new/enlarging T2 lesion during the 6 months preceding the enrollment;
- Steroids treatment during 30 days before the enrollment;
- Any contraindication to MRI;
- Patients currently participating in a different clinical trial;
- Patients currently pregnant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRIMRI will be performed every 3 months from baseline to 1 year. | Diagnostic test | Adult patients with NMOSD |
| Immunological FactorsImmunological factors will be performed every 3 months from baseline to 1 year. | Diagnostic test | Adult patients with NMOSD |
Primary outcome
Identifying predictors of clinical relapse
Time frame: From enrollment to 12 months
The primary outcomes measures are: percentage of patients with relapse, defined by the occurrence of a novel/worsening NMOSD symptom (detected by patient reported outcomes or clinical judgment or bioengineering indexes) PLUS biological findings of astrocyte damage (increase of GFAP level and/or MRI water index).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Background: PubMed 34362853Masuda H, Mori M, Hirano S, Uzawa A, Uchida T, Muto M, Ohtani R, Aoki R, Kuwabara S. Silent progression of brain atrophy in aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder. J Neurol Neurosurg Psychiatry. 2022 Jan;93(1):32-40. doi: 10.1136/jnnp-2021-326386. Epub 2021 Aug 6.
- Background: PubMed 41627519Margoni M, Gueye M, Meani A, Pagani E, Valsasina P, Storelli L, Preziosa P, Moiola L, Rocca MA, Filippi M. Substrates of 8.5-year clinical outcomes in aquaporin-4 IgG-positive neuromyelitis optica spectrum disorders. J Neurol. 2026 Feb 2;273(2):112. doi: 10.1007/s00415-026-13647-x.
- Background: PubMed 40506118Lorefice L, Carotenuto A, Fenu G. Silent burden: recognising and managing invisible symptoms in neuromyelitis optica. J Neurol Neurosurg Psychiatry. 2025 Jul 16;96(8):744-752. doi: 10.1136/jnnp-2025-336041.
- Background: PubMed 35483915Cacciaguerra L, Pagani E, Radaelli M, Mesaros S, Martinelli V, Ivanovic J, Drulovic J, Filippi M, Rocca MA. MR T2-relaxation time as an indirect measure of brain water content and disease activity in NMOSD. J Neurol Neurosurg Psychiatry. 2022 Apr 28:jnnp-2022-328956. doi: 10.1136/jnnp-2022-328956. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuromyelitis Optica Spectrum Disorders, run by IRCCS San Raffaele at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Massimo Filippi, Prof. (+39-02-26433032, filippi.massimo@hsr.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identifying predictors of clinical relapse (time frame: From enrollment to 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07526298, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026; AzTrials last refreshed its copy on 7 Nov 2026.